Rezolute Announces FDA Recommendation to Hold a Pre-BLA Meeting as a Precursor to Submitting a Biologics License Application (BLA) for Congenital Hyperinsulinism
Globe Newswire•05/10/2026•07:00 ET
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Key Highlights
- ➤FDA recommended a pre-BLA meeting for congenital HI despite sunRIZE endpoint failures
- ➤sunRIZE reduced hypoglycemia events, but primary SMBG endpoint missed statistical significance
- ➤Rezolute expects topline Phase 3 upLIFT tumor HI results this quarter
- ➤Company will assess potentially combined BLA strategy after upLIFT results
- ➤FDA made no determination on ersodetug’s approvability for congenital HI
Expert Statements
Nevan Charles Elam, Chief Executive Officer and Founder of Rezolute
“We appreciate FDA’s thorough review of the extensive body of data generated in sunRIZE and are encouraged by the Agency’s recognition that this data will require in-depth evaluation during a formal BLA review.”
Nevan Charles Elam, Chief Executive Officer and Founder of Rezolute
“We share FDA’s commitment to addressing the significant unmet needs of individuals living with rare diseases like congenital HI, where traditional drug development can present unique challenges.”
Nevan Charles Elam, Chief Executive Officer and Founder of Rezolute
“We believe this unusual process of preliminary review of detailed data demonstrates what can be achieved when regulators, companies, clinicians, and advocacy organizations work collaboratively to evaluate the totality of evidence for promising rare disease therapies.”
Julie Raskin, Chief Executive Officer of Congenital Hyperinsulinism International (CHI)
“For many people living with congenital hyperinsulinism, the threat of severe and unpredictable hypoglycemia remains a daily reality, and existing treatments are not enough.”
Julie Raskin, Chief Executive Officer of Congenital Hyperinsulinism International (CHI)
“We are encouraged by the progress toward potential new therapies and grateful to the patients, families, researchers, clinicians, regulators, and biotechnology companies working together to bring better options to our community.”
The Agency reiterated its commitment to addressing the unmet need for treatment of congenital and tumor-related hyperinsulinism and to serving these patient communities
On track to report topline results for the Phase 3 upLIFT study in tumor HI this quarter
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